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Baxter Announces Approval of ADVATE for Prophylaxis and Longer-Acting rFVIII Trial

January 1, 2012

Baxter International, Inc., announced in December 2011 that the U.S. Food and Drug Administration (FDA) has approved ADVATE, one of the company’s recombinant factor VIII products, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children (0-16 years) with hemophilia A.

The approval is based on a Phase IV, Baxter-sponsored study demonstrating that ADVATE for routine prophylaxis significantly reduced median annual bleed rates in hemophilia A patients from 44 to 1, compared to an on-demand regimen. In the study, 42% of patients had no bleeds during one year on prophylaxis.

“Emerging data provide important information to help physicians optimize care for hemophilia patients of all ages by preventing unexpected bleeding events that can have a detrimental impact on the lives of patients,” said Leonard Valentino, MD, Director, Rush Hemophilia and Thrombophilia Center and Section of Pediatric Hematology/Oncology, Rush University Medical Center, Chicago, and lead investigator of this study. “These data confirm the important clinical benefits of ADVATE when used as a prophylactic therapy to reduce bleeding episodes.”

ADVATE is also indicated for the control and prevention of bleeding episodes in adults and children (0-16 years) with hemophilia A, and perioperative management in adults and children (0-16 years) with hemophilia A.

Baxter also announced the dosing of the first patients in a Phase I clinical trial of its lead investigational candidate, BAX 855, a longer-acting (PEGylated) form of a full-length recombinant factor VIII (rFVIII) protein therapy. The manufacturing process for BAX 855, which uses rFVIII molecules and is free of plasma or albumin, is the same as that for ADVATE.

The Phase I trial is a prospective, open-label study that will assess the safety, tolerability and pharmacokinetics of BAX 855 in previously treated patients 12 years or older with severe hemophilia A. When used for prophylaxis, Baxter’s ADVATE requires patients to infuse every two to three days to reduce the occurrence of bleeding episodes.  This phase I trial is the first step in assessing whether BAX 855 can be infused less frequently.

“This trial is designed to provide new insights about our investigational longer-acting FVIII molecule, BAX 855, with the ultimate goal of improving care for patients living with hemophilia A,” said Hartmut J. Ehrlich, MD, vice president of global research and development in Baxter's BioScience business. “The Phase I results will serve as the foundation for advancing this important program through clinical development and determining whether BAX 855 can offer a treatment regimen requiring fewer infusions than ADVATE.”

Source: Baxter International, Inc.